SBIR Phase I: Development of a tolerizing platform for autoimmune diseases

The broader impact/commercial potential of this Small Business Innovation Research (SBIR) Phase I project is a novel therapeutic to treat early-stage (Stage 1 and Stage 2) type 1 diabetes, a significant and rapidly growing disease. The therapeutic will prevent insulin dependence and progression in type 1 diabetic patients to avoid more severe and chronic problems affecting their quality of life, overall medical burden and costs. This project may also enable development of therapeutics for other autoimmune diseases. This Small Business Innovation Research (SBIR) Phase I project will complete the design of a tolerizing therapeutic for type 1 diabetes by delivering epitopes via a single mRNA. The scope of activities include defining a panel of derived tolerizing epitopes including from the multiple proteins which drive the progression of type 1 diabetes, and aims to be effective in all type 1 diabetes patients. Selection of this panel of epitopes will allow the therapeutic to stop the pathogenic autoimmune reactions, to reduce or halt disease progression. The work in this project has two major activities: 1) in vitro binding analysis of the epitope peptides to disease relevant Major Histocompatibility Complex Class II haplotypes and 2) in vitro testing of the epitopes using cell lines and human cells. Each epitope in the panel will be evaluated to determine its Human Leukocyte Antigen binding profile to ensure binding to the disease associated proteins. The epitopes will each be tested against blood from type 1 diabetics and healthy donors to confirm reactivity of at least one epitope from each protein for each of the Human Leukocyte Antigen defined patient types to develop a robust and durable therapeutic. This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.